A therapy with miglustat, 2-hydroxypropyl-ß-cyclodextrin and allopregnanolone restores splenic cholesterol homeostasis in Niemann-pick disease type C1.

Anna-Maria NeßlauerAnne GläserMarkus GrälerRobby EngelmannBrigitte Müller-HilkeMarcus FrankChristine BursteinArndt RolfsJohn NeidhardtAndreas WreeMartin WittAnja Ursula Bräuer

Published in: Lipids in health and disease (2019)

In summary, the treatment promotes normal spleen morphology, stabilization of lipid homeostasis and blocking of inflammation, but alters the composition of T cell subtypes.

Keyphrases

oxidative stress
stem cells
fatty acid
ionic liquid
mesenchymal stem cells
low density lipoprotein
mass spectrometry
capillary electrophoresis
smoking cessation